Bluebird hit with FDA delays on gene therapies

Jan. 19, 2022

Bluebird Bio will have to wait a bit longer for the pending approval on two groundbreaking gene therapies, beti-cel and eli-cel, after the U.S. FDA extended the review period, the company announced.

The revised PDUFA goal dates for beti-cel and eli-cel are August 19, 2022, and September 16, 2022, respectively. The dates were extended to allow time to review additional information previously submitted. The information was deemed a major amendment but was not related to new safety events for either drug, said Bluebird in a press release.

“Gene therapies are complex, potentially transformative treatment options for those living with severe genetic diseases,” said Andrew Obenshain, CEO of Bluebird.

Bluebird, based in Massachusetts, will continue working with the FDA to review and bring these gene therapies to patients in the U.S. later this year, Obenshain said.

If approved, beti-cel and eli-cel would be the first lentiviral vector gene therapies for patients with severe genetic disease in the U.S. Both drugs have been granted Orphan Drug status, Breakthrough Therapy designation and Rare Pediatric Disease designation. The extended review is not expected to impact the priority review status of the drugs.

Beti-cel is a one-time gene therapy designed to add functional copies of a beta thalassemia cell to the blood of patients who require regular red blood cell transfusions. Eli-cel is for patients with cerebral adrenoleukodystrophy and aims to preserve as much neurological and motor function and communication ability as possible.

This delay comes following a hold the FDA placed on the company back in December. Bluebird’s trials for a gene therapy treatment called lovo-cel, intended to treat sickle cell disease, were hit with a clinical hold for patients under 18 following unexpected side effects during trial.

“We all share a responsibility to be diligent for patients as we progress in this novel field,” said Obenshain.